Eculizumab treatment in severe pediatric STEC-HUS: a multicenter retrospective study.

Détails

ID Serval
serval:BIB_D517DEE7C270
Type
Article: article d'un périodique ou d'un magazine.
Collection
Publications
Institution
Titre
Eculizumab treatment in severe pediatric STEC-HUS: a multicenter retrospective study.
Périodique
Pediatric nephrology
Auteur(s)
Percheron L., Gramada R., Tellier S., Salomon R., Harambat J., Llanas B., Fila M., Allain-Launay E., Lapeyraque A.L., Leroy V., Adra A.L., Bérard E., Bourdat-Michel G., Chehade H., Eckart P., Merieau E., Piètrement C., Sellier-Leclerc A.L., Frémeaux-Bacchi V., Dimeglio C., Garnier A.
ISSN
1432-198X (Electronic)
ISSN-L
0931-041X
Statut éditorial
Publié
Date de publication
08/2018
Peer-reviewed
Oui
Volume
33
Numéro
8
Pages
1385-1394
Langue
anglais
Notes
Publication types: Journal Article ; Multicenter Study
Publication Status: ppublish
Résumé
Hemolytic uremic syndrome related to Shiga-toxin-secreting Escherichia coli infection (STEC-HUS) remains a common cause of acute kidney injury in young children. No specific treatment has been validated for this severe disease. Recently, experimental studies highlight the potential role of complement in STEC-HUS pathophysiology. Eculizumab (EC), a monoclonal antibody against terminal complement complex, has been used in severe STEC-HUS patients, mostly during the 2011 German outbreak, with conflicting results.
On behalf of the French Society of Pediatric Nephrology, we retrospectively studied 33 children from 15 centers treated with EC for severe STEC-HUS. Indication for EC was neurologic involvement in 20 patients, cardiac and neurologic involvement in 8, cardiac involvement in 2, and digestive involvement in 3. Based on medical status at last follow-up, patients were divided into two groups: favorable (n = 15) and unfavorable outcomes (n = 18).
Among patients with favorable outcome, 11/14 patients (79%) displayed persistent blockade of complement activity before each EC reinjection. Conversely, in patients with unfavorable outcome, only 9/15 (53%) had persistent blockade (p = n.s.). Among 28 patients presenting neurological symptoms, 19 had favorable neurological outcome including 17 with prompt recovery following first EC injection. Only two adverse effects potentially related to EC treatment were reported.
Taken together, these results may support EC use in severe STEC-HUS patients, especially those presenting severe neurological symptoms. The study, however, is limited by absence of a control group and use of multiple therapeutic interventions in treatment groups. Thus, prospective, controlled trials should be undertaken.
Mots-clé
Acute Kidney Injury/etiology, Acute Kidney Injury/prevention & control, Antibodies, Monoclonal, Humanized/pharmacology, Antibodies, Monoclonal, Humanized/therapeutic use, Child, Child, Preschool, Complement Activation/drug effects, Complement Activation/immunology, Complement C5/antagonists & inhibitors, Complement C5/immunology, Complement Inactivating Agents/pharmacology, Complement Inactivating Agents/therapeutic use, Escherichia coli Infections/complications, Escherichia coli Infections/drug therapy, Escherichia coli Infections/immunology, Escherichia coli Infections/microbiology, Female, Follow-Up Studies, Hemolytic-Uremic Syndrome/complications, Hemolytic-Uremic Syndrome/drug therapy, Hemolytic-Uremic Syndrome/immunology, Hemolytic-Uremic Syndrome/microbiology, Humans, Infant, Male, Retrospective Studies, Severity of Illness Index, Shiga-Toxigenic Escherichia coli/isolation & purification, Treatment Outcome, Acute kidney injury, Complement, Hemolytic uremic syndrome, Pediatric
Pubmed
Web of science
Création de la notice
29/03/2018 18:14
Dernière modification de la notice
04/10/2019 5:09
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